“You don’t have to be a rocket scientist to know that the impact on Medicare is likely to be devastating,” said Diana Zuckerman, the president of the National Center for Health Research, who is not on the advisory committee that evaluated Biogen’s drug.
The $56,000 annual price tag of Biogen’s new drug, Aduhelm, already threatens to balloon health care costs — potentially swelling premiums for millions of older adults across the U.S. Patients could be on the hook for 20 percent of its cost. Or they could have supplemental insurance to cover the drug, but still might be responsible for thousands of dollars in fees for PET scans, which can confirm the presence of the amyloid plaque in the brain.
“Ideally, we want to use [Aduhelm] only on patients that we know have a reasonable chance of benefiting from that therapy,” Eric Rohren, chair of radiology at Baylor College of Medicine, said of the drug, which targets toxic protein buildup in the brains of people with the disease. “And so that’s where that role of PET scanning comes in: really identifying the patients with amyloid deposition who we predict will respond to the therapy.”
Patients receive the drug intravenously in a health care setting, meaning it falls under the purview of Medicare Part B. But in 2013, the Centers for Medicare and Medicaid Services decided that it generally won’t cover the specific PET scans used in Biogen’s clinical trials to help determine whether a patient has the amyloid buildups consistent with Alzheimer’s disease — and that’s putting pressure on the federal Medicare agency to cover the cost of making the diagnosis. States also have serious skin in the game because their Medicaid programs will feel pressured to cover the drug.
In September, Biogen, Eli Lilly and Company, GE Healthcare and Life Molecular Imaging requested that CMS reconsider its national coverage policy for PET in dementia and neurodegenerative disease.
“[Amyloid] PET imaging is reasonable and necessary for patients with cognitive impairment who are being evaluated for AD, including mild cognitive impairment due to AD, and who are being considered for treatment with an FDA-approved anti-amyloid therapeutic in a manner consistent with FDA labeling,” the companies wrote.
But CMS has not indicated whether it would reconsider its earlier National Coverage Determination — a process that would take months. The agency didn’t respond to repeated requests for comment on PET scans.
Susan Bunning, industry director at Medical Imaging and Technology Alliance, which represents imaging equipment manufacturers, told POLITICO the industry is hopeful CMS will immediately reconsider its 2013 decision that placed limitations on PET scan reimbursement now that the FDA has approved Aduhelm.
“Biogen has the ability to make this drug available much faster than providers will have the ability to provide it to their patients,” said Juliette Cubanski, a Kaiser Family Foundation deputy director. She listed several hurdles: the lengthy coverage decision process, the logistics of getting PET scans or the need to go to an infusion center.
It’s also not clear how Medicare would cover the newly approved Alzheimer’s therapy. CMS Administrator Chiquita Brooks-LaSure declined to say whether the agency would launch a process for determining how Medicare will cover the drug, which could install clinical guidelines that effectively narrow who’s eligible for the treatment.
“We are really focused on making sure that people have access to innovative drugs and can afford it,” Brooks-LaSure said at a press briefing Monday. “We’re considering our options, and we’ll be looking very closely at the science moving forward.”
Coverage from Medicare would undoubtedly mean higher premiums for 56 million older adults, and some experts say the federal government shouldn’t cover the drug given the scant evidence it works.
“As a private citizen, I think the drugs should not be covered by Medicare,” said Peter Bach, the director of the Center for Policy and Health Outcomes at Memorial Sloan Kettering. Bach chairs the Medicare coverage advisory committee, which could weigh in on the decision.
Meanwhile, private insurers haven’t said how they will cover the therapy. According to several insurance lobbyists, health plans are in the beginning stages of strategizing how they will approach coverage of Aduhelm and associated care.
“We need to prioritize both access and affordability, guided by input from experts based on clear clinical efficacy and scientific evidence,” David Allen, spokesperson for major insurer lobby AHIP, said in a statement.
Aduhelm works by eliminating amyloid, deformed proteins whose accumulation in the brain is thought to cause Alzheimer’s. But testing to specifically confirm its presence shakes up the standard diagnostic procedures for the condition. Medicare at the moment doesn’t cover PET scans to find amyloid. Finding it doesn’t impact care for people with Alzheimer’s with current treatments. “Up to this point, the diagnosis and care could be handled by a primary care doctor,” Halima Amjad, geriatrician at Johns Hopkins University, told POLITICO.
If a person sought help for worsening problems with memory or cognition, it’d be possible for a general practitioner to rule out other causes of dementia, like extra fluid, tumors, burst blood vessels or drug interactions in the brain. Even if a person didn’t definitively have Alzheimer’s, the care for most forms of dementia in its earlier phases is the same: A combination of the existing oral medications available to temporarily boost cognition as it declines.
But there’s substantial evidence that confirming the diagnosis of Alzheimer’s disease improves care substantially, said Gil Rabinovici, a neurologist and radiologist at the University of California San Francisco. After Medicare denied coverage of amyloid PET in 2013, Rabinovici and other colleagues conducted multiple studies that included 18,500 Medicare beneficiaries undergoing treatment for dementia.
The studies evaluated the potential benefit of new technologies, found that in about a third of all cases, confirming the presence of amyloid was worth it. “In our study the diagnosis changed after the PET scan in 35 percent of all people,” he said. Roughly 55 percent of participants with mild cognitive impairment, the earliest stages of dementia, had amyloid, as did 70 percent of people with dementia.
The other way to confirm the presence of amyloid is by looking for its presence in the fluid that surrounds the brain. Doing so requires a spinal tap, in which health care providers drain a trace amount of fluid.
“It’s a viable alternative,” said William Morice, the president of Mayo Clinic Laboratories. Biogen has partnered with Mayo Clinic Laboratories and Labcorp to provide more of these particular kinds of tests.
But lumbar punctures are invasive, impractical and imperfect.
“[A spinal tap] is probably not suitable for all patients,” said Anja Mett, neurology product leader of GE Healthcare’s pharmaceutical diagnostics business.
Practically, PET scans may be a better method for identifying good candidates for the drug. But Aduhelm’s labeling information is vague and doesn’t require patients to have confirmed amyloid at all. Health care providers will likely follow the protocol Biogen follows in a follow-up observational trial required to reassess the drug’s real-world efficacy. However, the company hasn’t yet pinned down the specifics of this trial yet.
“We are working on the final protocol of the confirmatory trial, but it is too early in the process to share more specifics,” Biogen spokesperson Allison Parks said in an email. “Details of the additional study will be provided at a later time.”
Some health care providers fear that aside from getting a concrete diagnosis, many patients won’t have physical access to the drug. Aduhelm requires a continuous IV; most primary care practices aren’t equipped with infusion centers, said Johns Hopkins’ Amjad. Even specialty memory care facilities, which are predominately in major cities and not rural areas, don’t have them; these are more common for cancer treatment.
Another concern is safety monitoring. In Biogen’s last clinical trials, some participants — particularly those with two copies of a gene known to increase the risk for developing Alzheimer’s disease — experienced a swelling or bleeding of the brain as the drug removed amyloid. This swelling and bleeding can be dangerous.
These individuals were in the earliest stages of Alzheimer’s, with relatively little amyloid. Because the FDA’s label for Aduhelm didn’t specify what stage of disease it was for, some physicians have concerns that this side effect may be worse for more advanced patients.
Practically, health care providers will need to monitor brain health with an MRI, which could be another logistical hurdle for patients and their caregivers. And demand could increase as more patients are eligible for the drug.
“The package insert specifically talks about MRI that patients need to have … within a year of starting the treatment, and then at various time points after initiation of therapy,” Rohren said. “And certainly if they have symptoms or side effects.”
“This will definitely put pressure on MRI systems to be able to accommodate the new patients coming in if we do see this therapy take off,” he said.
Researchers are looking toward other kinds of tests that could detect amyloid in the brain. Of particular interest are blood tests that could detect proteins that correlate with amyloid in the brain. These tests are appealing because they can be administered at any kind of health care facility — but they’re still years away from widespread adoption.
“I think the fact that this has now received FDA approval will certainly spur a lot of innovation around blood-based biomarkers for dementia and other diseases,” said the Mayo Clinic’s Morice.
Richard Hodes, the director of the National Institute on Aging, told POLITICO that blood biomarker tests could be used in the future to determine whether individuals should seek a PET scan to confirm the presence of amyloids in the brain.
The availability of a novel treatment for Alzheimer’s will inevitably garner significant demand — even though the drug’s benefits for all people living with the disease are unclear. But the sheer cost of the drug has huge implications for the American health care system’s financial viability — and could sock taxpayers with the cost.
The FDA is no stranger to public debate about its regulatory decisions, but its approval of Biogen’s drug has the potential to spark an even larger controversy than when it gave the greenlight to Sarepta Therapeutics’ duchenne muscular dystrophy drug Exondys 51 in 2016.
“Sarepta’s drug was for a rare disease; this is for a huge number of people,” Zuckerman said. “Don’t we all know someone who is going to use this drug?”
Sarah Owermohle and Susannah Luthi contributed to this report.